Gene therapy is transforming lives, but for many Americans it's hard to reach

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

Jaguar Gene Therapy completes Cohort I patient dosing in JAG201 clinical trial

Clinical-stage biotechnology company Jaguar Gene Therapy has completed dosing in Cohort I of its first-in-human clinical ...

First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

After a baby became the world's first CRISPR gene editing therapy patient, CHOP wants to expand treatment to others

One year after receiving CRISPR gene editing therapy at CHOP, a Delaware County baby is thriving, and doctors want to expand ...

Engineered protein markers read living brain gene activity in monkeys via blood

Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, ...
Fierce Pharma

BioMarin pulls hemophilia gene therapy Roctavian, taking $240M hit after divestiture efforts flounder

Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and ...
Fierce Biotech

Boehringer axes inhaled gene therapy after viewing cystic fibrosis data

Boehringer Ingelheim is discarding an inhaled gene therapy designed to treat cystic fibrosis (CF) after terminating a phase 1/2 trial. | Boehringer Ingelheim is discarding an inhaled gene therapy ...
Fierce Biotech

After gene therapy exit, Pfizer locks in global license for Beam gene editing candidate

After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.

Neurogene: Stock Soars On FDA Designation For Rett Gene Therapy - I'd Remain Cautious

Neurogene received FDA Breakthrough Therapy Designation for NGN-401 in Rett Syndrome, driving a >25% share price surge. Click ...
NC Biotech

Precision BioSciences cleared to begin gene therapy trial for muscular dystrophy

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

Hope And Hurdles For Sickle Cell Gene Therapy

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

Why Regeneron Pharmaceuticals (REGN) is One of the Top Gene Therapy Stocks to Buy According to Hedge Funds

Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) is one of the top gene therapy stocks to buy according to hedge funds.